A technique which allows genetic editing has been successfully tested on human cells meaning that genetic medicine -- previously an expensive and complex endeavour -- could become simple and affordable.
The process was discovered in 2012 by Jennifer Doudna and Martin Jinek of the Howard Hughes Medical Institute at the University of California, Berkeley working with Emmanuelle Charpentier of the Laboratory for Molecular Infection Medicine Sweden. Initially it described genomic editing in bacterial cells but has now been tested on human DNA .
"The ability to modify specific elements of an organism's genes has been essential to advance our understanding of biology, including human health," said Doudna. "This is going to remove a major bottleneck in the field, because it means that essentially anybody can use this kind of genome editing or reprogramming to introduce genetic changes into mammalian or, quite likely, other eukaryotic systems."
The editing system uses an enzyme-RNA complex, Cas9, present in some bacteria which conduct genetic editing for self preservation. The bacteria can cut up viral DNA strands and integrate it with their own DNA. They are then able to synthesise working copies of the genetic material in the form of RNA which binds to and inactivates the attacking virus.
"The beauty of this compared to any of the other systems that have come along over the past few decades for doing genome engineering is that it uses a single enzyme," explained Doudna. "The enzyme doesn't have to change for every site that you want to target -- you simply have to reprogram it with a different RNA transcript, which is easy to design and implement."
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Editing tool successfully created for human genome